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An open-label extension study evaluating the safety and efficacy of romiplostim for up to 3.5 years in thrombocytopenic Japanese patients with immune thrombocytopenic purpura (ITP)

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Abstract

Long-term use of the thrombopoietin mimetic romiplostim was examined in Japanese patients with chronic immune thrombocytopenic purpura (ITP) in this open-label extension. The starting dose of romiplostim was the previous trial dose or 3 μg/kg/week, which was titrated up to 10 μg/kg/week to maintain platelet counts between 50 and 200 × 109/L. As of April 2010, 44 patients had enrolled; 71 % women, median age 55.5 years, with five patients discontinuing romiplostim due to patient request (2), administrative decision (2), or not achieving study-defined platelet response (1). Median treatment duration was 100 weeks; median average weekly dose was 3.8 μg/kg. Twenty-eight patients (64 %) self-injected romiplostim. The most frequent adverse events were nasopharyngitis and headache. Nine patients (20 %) had a total of 14 serious adverse events (0.31/100 patient-weeks); of these, only oral hemorrhage was considered treatment related. Fifty hemorrhagic adverse events were reported in 20 patients (46 %) (1.12/100 patient-weeks). Ninety-six percent of patients had a platelet response (doubling of baseline platelet count and platelet count ≥50 × 109/L). Of the 25 patients receiving concurrent ITP therapy at baseline, all reduced or discontinued the therapy. Eight patients (18 %) received rescue medications. Administration of up to 3.5 years of romiplostim increased platelet counts and was well tolerated in Japanese patients with chronic ITP.

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Acknowledgments

The authors would like to thank all the study investigators and study coordinators, as well as Dr. Kiyoshi Kitamura of Tokyo University who provided medical advice during the study. This study was supported by Amgen Inc and Kyowa Hakko Kirin Co., Ltd. Medical writing assistance was provided by Susanna Mac, MD/PhD, of Amgen Inc.

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Authors and Affiliations

  1. Department of Hematology and Oncology, Tokai University Hospital, Kanagawa, Japan

    Yukari Shirasugi & Kiyoshi Ando

  2. Department of Hematology, Kitasato University Hospital, Kanagawa, Japan

    Koji Miyazaki

  3. Department of Blood Transfusion, Osaka University Hospital, Osaka, Japan

    Yoshiaki Tomiyama

  4. Department of Blood Transfusion, Hiroshima Red Cross Hospital and Atomic-bomb Survivors Hospital, Hiroshima, Japan

    Koji Iwato

  5. Division of Hematology, Keio University Hospital, Tokyo, Japan

    Shinichiro Okamoto

  6. Department of Hematology and Oncology, University of Tokyo Hospital, Tokyo, Japan

    Mineo Kurokawa

  7. Department of Hematology and Oncology, University of Yamanashi Hospital, Yamanashi, Japan

    Keita Kirito

  8. Department of Gastroenterology and Hematology, Hokkaido University Hospital, Hokkaido, Japan

    Satoshi Hashino

  9. Division of Hematology, Tsukuba University Hospital, Ibaraki, Japan

    Haruhiko Ninomiya

  10. Department of Hematology and Oncology, Kansai Medical University Hirakata Hospital, Osaka, Japan

    Shinichiro Mori

  11. Department of Transfusion Medicine and Cell Therapy, Kumamoto University Hospital, Kumamoto, Japan

    Yuji Yonemura

  12. Division of Hematology, NTT Kanto Medical Center, Tokyo, Japan

    Kensuke Usuki

  13. Amgen Inc., Thousand Oaks, CA, USA

    Helen Wei & Richard Lizambri

Authors
  1. Yukari Shirasugi
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  2. Kiyoshi Ando
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  13. Kensuke Usuki
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  15. Richard Lizambri
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Correspondence to Yukari Shirasugi.

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Shirasugi, Y., Ando, K., Miyazaki, K. et al. An open-label extension study evaluating the safety and efficacy of romiplostim for up to 3.5 years in thrombocytopenic Japanese patients with immune thrombocytopenic purpura (ITP). Int J Hematol 95, 652–659 (2012). https://doi.org/10.1007/s12185-012-1065-2

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  • DOI: https://doi.org/10.1007/s12185-012-1065-2

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